A randomized, controlled, delayed start trial of GM1 ganglioside in treated Parkinson's disease patients

J Neurol Sci. 2012 Nov 28. pii: S0022-510X(12)00581-3. doi: 10.1016/j.jns.2012.10.024. [Epub ahead of print]

Schneider JS, Gollomp SM, Sendek S, Colcher A, Cambi F, Du W.

Source

Dept. of Pathology, Anatomy and Cell Biology and Parkinson's Disease Research Unit, Thomas Jefferson University, Philadelphia, PA 19107, USA.

Abstract

The present single center, double-blind, delayed start study was conducted to examine possible symptomatic and disease-modifying effects of GM1 ganglioside in Parkinson's disease (PD). Seventy-seven subjects with PD were randomly assigned to receive GM1 for 120weeks (early-start group) or placebo for 24weeks followed by GM1 for 96weeks (delayed-start group). Washout evaluations occurred at 1 and 2years after the end of treatment. Seventeen additional subjects who received standard-of-care were followed for comparative information about disease progression. Primary outcome was change from baseline Unified Parkinson's Disease Rating Scale (UPDRS) motor scores. At week 24, the early-start group had significant improvement in UPDRS motor scores vs. a significant worsening of scores in the delayed-start group. The early-start group also showed a sustained benefit vs. the delayed-start group at week 72 and at week 120. Both groups had significant symptom worsening during washout. This study provides evidence that GM1 use for 24weeks was superior to placebo for improving motor symptoms and that extended GM1 use (up to 120weeks) resulted in a lower than expected rate of symptom progression. The data from this small study suggest that GM1 may have symptomatic and potentially disease modifying effects on PD.